NL-OMON56459招募中不适用
A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1 - ACHIEVE study
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 64（—）
入选标准
- •1. Age 18 to < 50 years, at the time of signing the informed consent.
- •2. Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat
- •size > 100.
- •3. Age of onset of DM1 muscle symptoms >= 12 years
- •4. Clinically apparent myotonia equivalent to hand opening time of at least 2
- •seconds in the opinion of the Investigator
- •5. Hand grip strength and ankle dorsiflexion strength
- •a. Hand grip strength averaged from both sides >= 20% and <= 80% (± 5%) predicted
- •for age, sex, and height at screening
- •b. Ankle dorsiflexion strength averaged from both sides >= 20% and <= 80% (±10%)
- •predicted for age, sex, and height at screening.
- •Note: two sets of functional assessments must be performed during the Screening
- •period. Participants must meet inclusion criterion #5 on both sets of
- •functional assessments for study eligibility.
- •6. Able to complete 10MWT, stair ascend/descend, and 5×STS at screening without
- •the use of assistive devices such as canes, walkers, or orthoses. The use of
- •submalleolar orthoses and inserts or supports that do not extend above the
- •malleolus are permitted during testing
- •7. Body mass index (BMI) < 35kg/m2
- •8. If being treated with testosterone, on a stable replacement dose for 30 days
- •prior to screening
- •9. Participants must agree to follow protocol-specified contraception guidance
- •as described in Section 10.4.
- •10. Female participants must not be pregnant or breastfeeding
- •11. Capable of giving signed informed consent as described in Section 10.1.3 of
- •the protocol, which includes compliance with the requirements and restrictions
- •listed in the informed consent form (ICF) and in the protocol
- •12. Willingness and ability of participant to comply with and tolerate
- •scheduled visits, dosing
- •administration plan, and study assessments, including multiple needle muscle
- •procedures over the duration of the study
排除标准
- •1. Previous or ongoing medical condition, medical history, physical findings,
- •or laboratory abnormalities that in the opinion of the Investigator could
- •affect safety, make it unlikely that dosing schedule and follow-up will be
- •correctly completed, and/or impair the assessment and interpretation of study
- •2. History of major surgical procedure within 12 weeks prior to the start of
- •investigative product administration or an expectation of a major surgical
- •procedure (eg, implantation of cardiac defibrillator) during course of the
- •3. History of anaphylaxis
- •4. History of clinically significant liver disease or ongoing treatment for
- •liver disease
- •5. History of clinically significant hematologic disease or have any of the
- •following hematologic results at Screening: platelets or hemoglobin below the
- •lower limit of normal for age and sex.
- •6. History of clinically significant kidney disease, ongoing treatment for
- •kidney disease (treatment for hypertension is permitted) or estimated
- •glomerular filtration rate (eGFR) < 60 mL/min as calculated with the Chronic
- •Kidney Disease Epidemiology Collaboration (CKD-EPI) Cystatin C Equation (Inker
- •et al. 2012) at screening
- •7. Active malignancy or history within the last 5 years, except for basal or
- •squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has
- •been successfully treated
- •8. Recent history (within previous 12 months) of drug or alcohol abuse
- •9. Medical condition other than DM1 that would significantly impact ambulation
- •or participation in functional assessments
- •10. Current insulin-dependent diabetes mellitus or uncontrolled diabetes
- •mellitus, congestive heart failure, symptomatic cardiomyopathy, symptomatic
- •coronary artery disease, multiple sclerosis, or other serious medical illness
- •11. Second- or third-degree heart block, symptomatic first-degree heart block,
- •atrial flutter, atrial fibrillation, ventricular arrhythmias, pacemakers,
- •implanted defibrillator, or is receiving medication for treatment of cardiac
- •12. Treatment with medications that can improve myotonia of clinical functional
- •endpoints within a period of 5 half-lives of the medication prior to performing
- •screening assessments. May include but not limited to mexiletine, phenytoin,
- •carbamazepine, procainamide, disopyramide, ranolazine, flecainide, lamotrigine,
- •nifedipine, acetazolamide, clomipramine, imipramine, amitriptyline, taurine,
- •quinine, or metformin.
- •13. Use of anticoagulant such as warfarin or a direct oral anticoagulant (eg,
- •dabigatran) due to the increased risk of bleeding
- •14. Current treatment with immunosuppressive therapy
- •15. Receipt of another investigational drug, biologic agent, or device within 5
- •half-lives (if known) of the agent, or within 4 months prior to the start of
- •Screening, whichever is longer. Individuals previously treated with
- •oligonucleotide therapies (including small interfering RNA [siRNA]) may be
- •eligible if the last dose of the investigational drug was received >= 3 years ago
- •16. ECG with the corrected QT interval by Fridericia*s Formula (QTcF) >= 450 ms
- •in men and QTcF >= 460 ms in women, PR >= 240 ms, left bundle-branch block, or a
- •conduction defect, which is clinically significant in the opinion of the
- •Investigator
- •17. Percent predicted forced vital capacity (F
研究者
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